Egetis Therapeutics (EGTX) Status Update summary

EMA marketing authorisation plan

• Available Triac Trial I and published long-term data considered sufficient for an Emcitate MAA in Europe following regulatory interactions; required clinical data are already available, reducing remaining development risk.

• Revised EMA filing timelines to follow confirmation of the regulatory dossier; discussions with other authorities are ongoing, while Triac Trial II continues to study early intervention and neurocognitive development.

Clinical evidence

• Real-world cohort included 67 patients treated for a median 2.2 years, with treatment extending up to 6 years; serum T3 fell from 4.58 to 1.66 nmol/L from baseline to last visit (p<0.0001).

• Long-term findings confirmed Triac Trial I results, with T3 normalization and sustained or improving effects across clinical and biochemical measures, regardless of age.

• Triac Trial I enrolled 46 patients across 9 countries; the Phase II study reported significant, clinically relevant effects across secondary endpoints and was described as safe and tolerable.

Disease and ongoing study

• MCT8 deficiency is an X-linked disorder affecting males, with estimated prevalence of 1 in 70,000; it causes severe neurodevelopmental disability and has no available treatment addressing the disorder’s root cause.

• Triac Trial II is an early-intervention study in young patients; recruitment was progressing well and was expected to complete in Q1 2022.